AstronauTx is a Dementia Discovery Fund-formed company targeting astrocytes as the basis for treatments for dementias. It is the first spin-out company based on the work from the University College London Drug Discovery Institute (UDDI).

Astrocytes in Alzheimer's disease

Astrocytes are the most numerous cell type in the brain. These star-shaped cells are crucial support cells that have many important roles, from facilitating communication between nerve cells to maintaining blood supply in brain regions.

In Alzheimer’s disease, the shape and function of astrocytes are affected, leading to changes in cognition as the disease progresses.

Astrocytes schematic

What did the scientists do?

Scientists at the UDDI developed an "Astrocyte platform". This is a collection of experiments which they can be used to identify and test new drug targets in astrocytes.

The UDDI scientists are using this screening platform to identify the best possible molecule to block a protein and reduce astrocyte hyperactivity. The aim is to develop a new drug that can restore astrocyte function and normalise communication between nerve cells in Alzheimer’s disease.

 

So what?

This ‘Astrocyte platform’ attracted an investment of £6.5m from the Dementia Discovery Fund to form a new spin out company called AstronauTx.

AstronauTx later raised a further £1m from the UCL Technology Fund to continue their search for new drug targets for astrocytes in Alzheimer’s and other neurodegenerative diseases.

 

Testing the drug target P2Y1

The company worked with the UDDI to develop a lead molecule which was suitable for testing in mice models of Alzheimer’s disease. They used a technique that measures electrical activity of brains to show that the molecule changed brain activity in mice with features of Alzheimer’s disease.

This project reached the in vivo testing stage. Unfortunately, the drug was found to be toxic in rats, so the project has now been terminated.

 

Testing a drug to modulate PAC1 activity

PAC1 is a receptor that is predominately found in astrocytes. The activation of PAC1 is important for astrocyte function.

In Alzheimer’s disease, PACAP, the peptide that activates PAC1, has been shown to be reduced.

Scientists from the UDDI hypothesised that a drug that enhances PAC1 activity might restore the normal function of the astrocytes.

The UDDI has received £1.25M to work with AstronauTX to conduct large screening efforts for molecules that affect PAC1 function. They have tested thousands of molecules and have found a number of possible “hits”. The UDDI will refine these compounds to turn the “hits” into potential drugs.

Applications

The DDF is keen to hear from scientists, researchers and companies developing novel therapies for dementia, who are in need of funding and help.